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Huge study reveals new 'genetic modifiers' of Huntington's disease
GeM-HD identifies the first solid leads in the hunt for genetic variations that affect Huntington's disease onset
July 31, 2015
Important drug targets yielded by new genetic study of HD
A genetic study confirms that minute differences in DNA repair genes can influence the age of HD symptom onset.
May 02, 2016
An early role for the Huntington's disease gene – but don't believe all the headlines
A surprising new paper sheds light on the role of the HD gene early in development. Should we worry?
February 27, 2018
HDSA FAQ on the Roche/Genentech RG6042 program
HDSA's frequently asked questions factsheet about the huntingtin-lowering trial ASO program was so good we stole it (with permission)
October 19, 2018
Huntington's disease goes viral as UniQure inches ahead in gene therapy race
FDA grants “Investigational New Drug” status to Huntingtin-lowering gene-therapy agent AMT-130, clearing path to human trials in Huntington’s Disease patients
January 30, 2019
When interrupting is good: genetic hiccups that protect against Huntington's disease
Multiple teams find small differences in the 'CAG repeat' bit of the Huntington's disease gene. They don't directly change the huntingtin protein, but do alter the age of symptom onset. What's behind this enigma and what does it mean for patients?
March 14, 2019
Details emerge of first Huntington’s disease gene therapy clinical trial
UniQure announces key details of its planned trial to assess the safety and ability of AMT-130 gene therapy to lower the problematic huntingtin protein using a ‘single-shot’ virus delivery system.
July 17, 2019
Caution urged for the use of gene-editing technology CRISPR
A recent series of studies on the gene-editing method CRISPR have raised concerns about the suitability of this technology for the treatment of genetic illnesses such as Huntington's disease
August 12, 2020
When genes are unstable: targeting somatic instability in HD
CAG repeats expand in some parts of the body and brain as people with HD get older, a phenomenon known as somatic instability. Learn more about how researchers are exploring somatic instability and DNA repair to design therapies for HD.
September 08, 2020
Good news from uniQure: gene therapy trial on track, and promising data in animals
The first group of 10 participants have been dosed in uniQure’s clinical trial of an HD gene therapy, and three new manuscripts describe safe, widespread huntingtin lowering in animals.
April 15, 2021
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